I recently joined a campaign from I AM ALS to tear down some of the barriers that exist between different neurological diseases. Over the course of this year I have gotten to know one of the group’s co-founders, Brian Wallach. Brian is a lawyer, father, husband and advocate who was diagnosed with ALS in November 2017. He was working as a federal prosecutor in the United States Attorney’s Office when he set off to find out as much about ALS as he could. His story and the sense of urgency he exudes has been a constant source of inspiration for me.
Joining this campaign is also Justine Fedak. Justine is an executive, wife, mother and advocate and was diagnosed with multiple sclerosis 18 years ago. She has been an advocate for MS for over a decade.
Recently I AM ALS put together the following video of us to help launch this initiative. It was also featured as part of an interview of Brian and his wife Sandra on WGN, the largest TV station in Chicago.
But does it really make sense to lump these diseases together? Could new therapies for one really help tackle another?
Last month I gave the following talk (audio only) to the resident neurologists at the University of Toronto. In it I gave a summary of what I think are the biggest problems that the Parkinson’s disease field faces and some of the things that need to be done to solve them.
(To download the slides from that talk click here –> Breaking Barriers in Neurodegeneration – Toronto)
We had a great discussion afterwards in which it quickly became clear that almost none of the problems I outlined are unique to PD. Here are some of the key problems identified:
- We don’t understand the biology driving these diseases very well.
- Each disease is poorly defined and in reality likely represents a spectrum of disorder.
- There are very few incentives in place to promote the kind of broad collaborative efforts needed to push research forward.
- Science is primarily driven by what publishers and grant reviewers want. This promotes a culture in which the needs of patients and society are not prioritized.
- Clinical trials, as currently constructed, are a terrible way to test medical hypotheses.
- Drug development is painfully slow and rife with inefficiencies.
- Patients have inadequate access to the care they need.
However, there is one critique of this campaign I have heard that I wanted to address.
Medicine itself is moving away from grouping patients into large disease clusters and towards precision therapies tailored towards the specific molecular abnormalities of small groups. So, why cluster all these diseases together just to break them up into even smaller bits?
To that I would say, precision medicine only truly becomes precise when we have clear molecularly (or at least genetically) defined diseases. We aren’t there yet with neurodegenerative diseases, to get there we will need to make progress in almost all of the areas mentioned above. Until we have those unique signatures we need to be disease agnostic and focus on a more general understanding of how biology works, what aging does to our biology, and the influence genetic variants and different environmental exposures have.
In addition, the tools and techniques developed to get us a better understanding of biology and disease cut across classically defined disease boundaries. Antisense oligonucleotides, gene delivery, iPS cells, and many more recent advances in biology have had applications across the disease spectrum. Also, as far as we know, many of these diseases have similar biological mechanisms at work, micriobiome imbalances, inflammation, cellular senescence, mitochondrial dysfunction, etc. etc. all pop up in a number of these diseases.
Finally, recent evidence suggests that the diagnostic criteria we have been using to separate these diseases do not match up with what the biology tells us.
So, since almost all of the problems faced are common across disease boundaries, it seems to make sense to team up and see if we can’t achieve more together.
Over the coming months, I AM ALS will grow this effort by bringing together advocates and organizations across neurological diseases to accelerate our fights to find cures for all.
You raise an interesting point regarding precision medicine and Parkinson’s. Personalized medical care is a great concept (assuming it is affordable, and you don’t have to wait 6 months for an appointment), but precision medicine is absolutely the wrong term to be used to describe it….at least in the context of Parkinson’s, where neurologists are more like mixologists when it comes to prescribing medication.
I mean no disrespect to these neurologists (or bartenders or mixologists for that matter, as they might also be offended by this comparison). Treating Parkinson’s is both and art and a skill, but, with our current understanding of the disease, it is most definitely not precise.
– Brett